Synageva BioPharma Corp., a privately held biopharmaceutical company that is developing therapeutic products for rare disorders, announced today that the company is supporting the Fourth International Rare Disease Day observance on February 28, 2011.
“Synageva is excited to join EURORDIS and NORD and other supporting organizations to recognize the importance of this day.”
"Rare Disease Day has created significant momentum for the awareness of often overlooked but devastating conditions. Much progress has been made since the passage of the Orphan Drug Act of 1983, but many patients with rare diseases remain without effective treatment options," said Sanj K. Patel, President and CEO of Synageva BioPharma. "Synageva is excited to join EURORDIS and NORD and other supporting organizations to recognize the importance of this day."
Synageva has recently initiated the first ever global clinical program investigating an Enzyme Replacement Therapy (ERT) for Lysosomal Acid Lipase (LAL) Deficiency, a Lysosomal Storage Disorder (LSD). Also known as Wolman Disease, Cholesteryl Ester Storage Disease (CESD) or Acid Lipase Deficiency, LAL Deficiency is a rare, autosomal recessive, progressive disease with devastating consequences for infants, children and adults. In addition to this clinical program, Synageva has a pipeline of orphan therapeutics in development including two enzyme replacement therapies (ERTs) for Lysosomal Storage Disorders (LSDs) and two non-LSD programs for life-threatening conditions.